FDA Updates

The approval of irinotecan liposome offers an improved first-line treatment option for individuals diagnosed with metastatic pancreatic adenocarcinoma.

Iloprost had also received Priority Review and Orphan Drug Designations for this indication, and FDA approval in 2004 for the treatment of pulmonary arterial hypertension.

Repotrectinib led to a durable anti-tumor response in patients with NTRK-positive locally advanced or metastatic solid tumors.

If approved, this would mark the first MDMA-assisted therapy and psychedelic-assisted therapy approved, calling for a reschedule of MDMA from Schedule I. A PDUFA was set for this summer.

The updated indication for the respiratory syncytial virus vaccine is for adults aged 50 to 59 with underlying conditions who are at an increased risk of contracting the virus.

Bepirovirsen is a triple action investigational antisense oligonucleotide and is being evaluated in the B-Well phase 3 clinical trial program for the treatment of chronic hepatitis B.

The FDA approval marks the first and only FDA-approved oral therapy for this patient population, and the drug is expected to be available by the end of February.

In a study, nipocalimab helped more than 50% of patients with high-risk of hemolytic disease of the fetus and newborn achieve a safe live birth.

The FDA assigned a Prescription Drug User Fee Act (PDUFA) goal date of October 8, 2024.

In November 2023, the FDA announced an investigation of CAR T-cell therapy based on several reported secondary T-cell malignancies.

The company seeks the approval for both the intravenous and subcutaneous administration routes.

In December 2023, the FDA granted expanded indications for bempedoic acid and bempedoic acid plus ezetimibe in the treatment of primary hyperlipidemia.

Daratumumab and hyaluronidase-fihj in combination with other therapies was previously approved by the FDA in May 2020 for 8 indications in multiple myeloma.

Afami-cel had engineered t-cell receptor T-cells target MAGE-A4+ tumors, which are highly expressed in synovial sarcoma with HLA-A*02.

The designation offers support in developing potential new medicines, treatment, and diagnosis to prevent rare conditions, like EoE.

Less manufacturing time could be the difference between a patient receiving this life-saving therapy vs not.

Trastuzumab deruxtecan and elacestrant represent important new treatment options.

The approval marks dupilumab as the first and only FDA-approved medicine to treat pediatric patients with eosinophilic esophagitis.

According to the FDA review, patients with CKD receiving denosumab for osteoporosis who are on dialysis or have CKD-MBD are at the highest risk of severe hypocalcemia.

The treatment is for patients with KRAS G12C-mutant metastatic NSCLC who have previously received at least 1 systemic therapy and were not treated with a KRAS G12C inhibitor.

PTX-252 is a novel molecular drug designed to increase cancer cells' sensitivity to chemotherapy in the treatment of acute myeloid leukemia.

Previously, IgG-hyaluronidase recombinant was approved by the FDA in 2014 for the treatment of primary immunodeficiency in adult patients and has expanded to include some pediatric patients.

The approval was based on the results of a phase 3 trial that found an improvement in progression-free survival among individuals treated with pembrolizumab.

BrainSee creates a new standard for progression prediction in Alzheimer disease, marking a step forward in brain health management with non-invasive screening.

The accepted sBLA comes after phase 3 clinical trial results which demonstrated better overall survival, progression-free survival, and confirmed overall response rate than chemotherapy in patients with cervical cancer.

Teneo Excimer Laser Platform is a new laser assistance in situ keratomileusis for vision correction surgery for those who have myopia and myopic astigmatism.

Berdazimer topical gel, 10.3% (Zelsuvmi; Ligand Pharmaceuticals Inc) has been approved for the treatment of molluscum contagiosum in individuals aged 1 year and older.

Pharmacists' expertise is crucial in educating patients about regulatory changes, helping them navigate alternative formulations, and providing insights into evidence-based choices.

Adalimumab-aqvh (Yusimry; Coherus Biosciences) entered the market in July 2023, following approval by the FDA in December 2021.

These drugs have interesting mechanisms, unique use, or may support expanded indications in the future.